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Latest News: Research

What Is Happening About Access To Nusinersen?

18 October 2017 / Posted in: Research

We share the frustrations of the SMA Community that there has still been no announcement from NICE about whether it will assess the treatment for continued and wider provision of nusinersen by the NHS. Here is an update of what SMA Support UK is doing.

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AveXis to Imminently Initiate AVXS-101 Pivotal Trial in the US

12 October 2017 / Posted in: Research

The Food and Drug Administration (FDA) has granted permission to AveXis to begin their planned US pivotal trial of their gene therapy drug for SMA, AVXS-101. Avexis say that they are to immediately begin the trial.

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Join MDUK's 'FastTrack' Campaign

11 October 2017 / Posted in: Research

Muscular Dystrophy UK are campaigning for faster access to specialist treatments, including Spinraza for SMA. You can find out more and support the 'FastTrack' Campaign here.

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NHS England replies to the joint clinicians/charities letter

29 September 2017 / Posted in: Research

The request to include those with more than two SMN2 copies and over 7 months of age in the SMA Type 1 nusinersen EAP is not yet successful. We are still waiting to hear from NICE about next steps.

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Novartis Releases Clinical Trial Update

27 September 2017 / Posted in: Research

Novartis has released the following community update for the LMI070 (branaplam) clinical trial.

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Roche’s SUNFISH study community update

19 September 2017 / Posted in: Research

Roche have provided an update on clinical trials of their SMN2 splice-modifying drug RG7916

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Revisiting the Consensus Statement for Standards of Care in SMA - Full Report

30 August 2017 / Posted in: Research

You can now read the full workshop report written by the clinical researchers and patient representatives from Europe and the US who met last year.

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Letter to NHS England Requesting Review of EAP Type 1 Interim Policy Criteria

24 August 2017 / Posted in: Research

Sent on 23rd August from clinicians and advocacy groups, this asks for the age limit and SMN2 copy number restrictions on potential access to be removed.

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RG7916 FIREFISH trial now enrolling patients (though not in UK)

16 August 2017 / Posted in: Research

Roche has begun to enrol SMA patients in the FIREFISH Phase II trial of their splice-modifying drug, RG7916 (also known as RO7034067).

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Age of children who will be able to access the EAP for SMA Type 1 going forwards

07 August 2017 / Posted in: Research

We have clarified that, from now on going forwards (until we hear from NICE), children will have to be diagnosed by 7 months of age to be eligible. If you have a child who has already been diagnosed with SMA Type 1 who is older than this and not already on the EAP, please read more.

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